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FDA Approves 'Breakthrough' Leukemia Drug

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WebMD Health News

May 10, 2001 (Washington) -- In record time, the FDA approved Gleevec for the treatment of a rare but deadly type of leukemia. This pill for chronic myeloid leukemia, or CML, represents a new approach to controlling this disease -- and potentially many other cancers -- because it zeros in on cancer cells, leaving the normal ones alone.

"[Gleevec] appears to change the odds dramatically for patients, and it does so with relatively low occurrence of side effects. ... This oral drug is based on the concept of molecular targeting -- and we believe such targeting is the wave of the future," said Health and Human Services Secretary Tommy Thompson at a news conference here Thursday.

Some of Gleevec's more common side effects include nausea and other abdominal problems.

In CML, white blood cells grow out of control, eventually driving out oxygen-carrying red blood cells. The patient then develops chronic anemia and wastes away. About 4,500 Americans per year develop the condition, which is often untreatable in its later stage.

Currently, CML patients are treated with the medication interferon alfa, but it only works in some patients and can have serious side effects. Bone marrow transplantations can cure CML, but many patients aren't able to find a donor match or deal with the life-threatening side effects. In fact, several patients die from the transplantation itself.

For the last two years, researchers have been studying Gleevec as a way to specifically target and interfere with how CML works at its molecular level. The drug shuts down a key protein responsible for CML's abnormal cell growth.

"This single drug is as interesting and impressive as any we have ever seen throughout our long war on cancer. Yet there are many questions to be answered," says Richard Klausner, MD, director of the National Cancer Institute. Among the issues -- the long-term benefits and side effects of the drug. However, Klausner considers Gleevec "the picture of the future of cancer treatment."

Why?

Early studies carried out by Brian Drucker, MD, director of the Oregon Cancer Institute in Portland and one of Gleevac's primary developers, showed that patients who had failed virtually every other therapy responded to this treatment.

"Certainly, I don't think that anything has been seen like this before," Dawn Willis, PhD, scientific program director of the American Cancer Society, tells WebMD.

From that positive result just two years ago, the FDA went on to grant the drug's manufacturer, Novartis, a priority review. Then in just over two months Gleevec was given accelerated approval, a record time for a cancer drug.

Now Gleevec is being heralded as the first example of so-called "rational drug design" aimed at a targeting the genetic malfunction that exists in CML patients. In these individuals, an abnormal chromosome instructs the body to produce too much of a particular protein, which in turn sends out a message to produce more and more white cells.

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